Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study

OBJECTIVE: Gaucher disease (GD) is a lysosomal storage disease characterized by a deficiency of glucocerebrosidase. Although enzyme‐replacement and substrate‐reduction therapies are available, their efficacies in treating the neurological manifestations of GD are negligible. Pharmacological chaperon...

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Detalles Bibliográficos
Autores principales: Narita, Aya, Shirai, Kentarou, Itamura, Shinji, Matsuda, Atsue, Ishihara, Akiko, Matsushita, Kumi, Fukuda, Chisako, Kubota, Norika, Takayama, Rumiko, Shigematsu, Hideo, Hayashi, Anri, Kumada, Tomohiro, Yuge, Kotaro, Watanabe, Yoriko, Kosugi, Saori, Nishida, Hiroshi, Kimura, Yukiko, Endo, Yusuke, Higaki, Katsumi, Nanba, Eiji, Nishimura, Yoko, Tamasaki, Akiko, Togawa, Masami, Saito, Yoshiaki, Maegaki, Yoshihiro, Ohno, Kousaku, Suzuki, Yoshiyuki
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4774255/
https://www.ncbi.nlm.nih.gov/pubmed/27042680
http://dx.doi.org/10.1002/acn3.292

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